Transplantation and Cellular Therapy最新文献

{"title":"Generosity of the Unrelated Stem Cell Transplant Donor: Moving from Hope to Reality While Overcoming Challenges to Deliver the Gift of Life","authors":"Steven M. Devine","doi":"10.1016/j.jtct.2025.01.033","DOIUrl":"10.1016/j.jtct.2025.01.033","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 59-62"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"RBC Alloimmunization in SCD: Chipping Away at the Iceberg of Implications","authors":"RCG Azbell , PC Desai","doi":"10.1016/j.jtct.2025.01.035","DOIUrl":"10.1016/j.jtct.2025.01.035","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 66-68"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143081019","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Letermovir for Prevention of Recurrent Cytomegalovirus in High-Risk Allogeneic Hematopoietic Cell Transplantation Recipients","authors":"Gyuri Han ,&nbsp;Anat Stern ,&nbsp;Yeon Joo Lee ,&nbsp;Yuxuan Li ,&nbsp;Parastoo B. Dahi ,&nbsp;Roni Tamari ,&nbsp;Boglarka Gyurkocza ,&nbsp;Ann A. Jakubowski ,&nbsp;Esperanza B. Papadopoulos ,&nbsp;Brian Shaffer ,&nbsp;Miguel-Angel Perales ,&nbsp;Karam M. Obeid ,&nbsp;Jo-Anne H. Young ,&nbsp;Genovefa A. Papanicolaou","doi":"10.1016/j.jtct.2024.12.010","DOIUrl":"10.1016/j.jtct.2024.12.010","url":null,"abstract":"<div><div>We evaluated letermovir (LTV) for secondary prophylaxis for cytomegalovirus (CMV) in allogeneic hematopoietic cell transplant recipients (HCT) at high-risk for CMV recurrence. This open-label study was conducted at Memorial Sloan Kettering Cancer Center and the University of Minnesota. Patients with clinically significant CMV infection (cs-CMVi) and ≥1 high-risk criteria for CMV who achieved viral suppression with standard CMV antivirals received LTV secondary prophylaxis for up to 14 weeks. The primary endpoint was cs-CMVi at week 14; secondary endpoints included LTV resistance, CMV end-organ disease (EOD), CMV-related death, and LTV-related adverse events at week 14. Thirty-six patients were analyzed (CMV seropositive, n = 33; T cell-depleted HCT, n = 25; cord blood allograft, n = 5). By week 14 post-transplantation, 5 patients met the primary endpoint of cs-CMVi, for a cumulative incidence of 14.9% (95% confidence interval, 2.6% to 27.1%). Four patients developed LTV breakthrough cs-CMVi (including 2 patients with confirmed LTV resistance). The remaining patient developed rebound cs-CMVi after premature discontinuation of LTV due to enrollment in a clinical trial. There were no cases of CMV EOD, CMV-related death, or LTV-related adverse events by week 14 or by week 24. Our data support that LTV secondary prophylaxis is safe and effective in high-risk HCT recipients.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 105.e1-105.e9"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neurotoxicity and Rare Adverse Events in BCMA-Directed CAR T Cell Therapy: A Comprehensive Analysis of Real-World FAERS Data","authors":"Moataz Ellithi ,&nbsp;Magdi Elsallab ,&nbsp;Matthew A. Lunning ,&nbsp;Sarah A. Holstein ,&nbsp;Smriti Sharma ,&nbsp;Jonathan Q. Trinh ,&nbsp;Jihyun Ma ,&nbsp;Marcela V. Maus ,&nbsp;Matthew J. Frigault ,&nbsp;Christopher R. D'Angelo","doi":"10.1016/j.jtct.2024.12.002","DOIUrl":"10.1016/j.jtct.2024.12.002","url":null,"abstract":"<div><div>Chimeric antigen receptor T (CAR T) cell therapies have emerged as a valuable treatment modality for patients with plasma cell disorders. As the population of patients receiving CAR T therapies grows, the identification and management of associated rare toxicities become increasingly crucial. This study aims to identify safety signals associated with commercial anti-B-cell maturation antigen (BCMA) CAR T therapies using the Food and Drug Administration Adverse Event Reporting System (FAERS). We performed a cross-sectional analysis of the adverse events (AE) reports associated with ciltacabtagene autoleucel (cilta-cel) and idecabtagene vicleucel (ide-cel), submitted to FAERS between January 2021 and December 2023. AE frequencies were summarized using descriptive statistics, and safety signals were explored by measuring the reporting odds ratio (ROR) compared to control groups. Among 4,472,782 unique FAERS reports, 1,496 involved BCMA-directed CAR-T therapies. AEs reported more frequently included immune-associated conditions and neurological disorders. Neurotoxicity associated with cilta-cel predominantly manifested as cranial nerve palsies, Parkinson's disease and parkinsonism, and acute and chronic polyneuropathies, while ide-cel neurotoxicity presented as confusion, disorientation, seizures, balance disturbances, and tremors. In cilta-cel reports, other safety signals included Guillain-Barre syndrome (ROR: 17.1, 95% CI 6.1 to 47.5), intracranial hemorrhage and cerebrovascular accidents (ROR: 2.9, 95% CI 1.8 to 4.8), Haemophilus infections (ROR: 34.2, 95% CI 11.8 to 98.9) and cytomegalovirus infections (ROR: 3.9, 95% CI 1.6 to 9.5). For ide-cel, new signals included parkinsonism (ROR: 13.7, 95% CI 5.5 to 34.5), acute and chronic sarcoidosis (ROR: 197.1, 95% CI 32.9 to 1180.1), ventricular arrhythmias, and cardiac arrest (ROR: 3.9, 95% CI 2.1 to 7.3). This analysis provides a comprehensive insight into the safety profiles of the commercial BCMA-directed CAR T therapies, underscoring the importance of vigilant post-marketing surveillance to mitigate potential risks.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 71.e1-71.e14"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142822711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lipid Levels Increase to the Normal Range After Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease","authors":"Jackie Queen ,&nbsp;Emily Limerick ,&nbsp;Neal Jeffries,&nbsp;Matthew M. Hsieh,&nbsp;Robert D. Shamburek,&nbsp;Courtney D. Fitzhugh","doi":"10.1016/j.jtct.2024.12.008","DOIUrl":"10.1016/j.jtct.2024.12.008","url":null,"abstract":"<div><div>Individuals with sickle cell disease (SCD) have a unique type of dyslipidemia characterized by low total cholesterol (TC), low low-density lipoprotein cholesterol (LDL-c), low high-density lipoprotein cholesterol (HDL-c), and normal triglycerides (TG). This lipid state is theorized to be cardioprotective against atherosclerosis. In SCD, hematopoietic cell transplant (HCT) offers a potentially curative therapy. Long-term survivors of HCT for hematologic malignancies are at increased risk for dyslipidemia and atherosclerosis long-term. The effects of HCT on SCD dyslipidemia are unknown. This retrospective cohort study characterizes lipid profiles at baseline and after nonmyeloablative allogeneic HCT for SCD. We analyzed data from 116 patients after nonmyeloablative HLA-matched sibling or haploidentical HCT for SCD at the NIH from 2009 to 2021. TC, HDL-c, LDL-c, and TG were collected pre-HCT, 1-year post-HCT, and annually thereafter. Data were analyzed using linear generalized estimating equation regression modeling. Successful HCT was associated with a rise in TC, LDL-c, and HDL-c and a decline in TG post-HCT. After HCT, previously low lipid levels increased to the normal range. These changes occurred within the first year of HCT and were maintained thereafter. In patients with graft failure, TC and LDL-c levels remain unchanged from their pre-HCT baseline. Sirolimus use for graft versus host disease prophylaxis was associated with higher TG levels. These findings suggest that SCD dyslipidemia resolves with reversal of the SCD phenotype. The normalization of lipid parameters suggests SCD patients are not at increased risk for atherosclerosis after successful HCT compared to their peers; further studies with longer follow-up are required.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 82.e1-82.e8"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865565","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-Versus-Host Disease Survivorship After Hematopoietic Cell Transplantation: Part I. Phases of Chronic GVHD, Supportive Care, and Systemic Therapy Discontinuation","authors":"Neel S. Bhatt ,&nbsp;Andrew C. Harris ,&nbsp;Lev Gorfinkel ,&nbsp;Katarzyna Ibanez ,&nbsp;Eric R. Tkaczyk ,&nbsp;Sandra A. Mitchell ,&nbsp;Stacey Albuquerque ,&nbsp;Tal Schechter ,&nbsp;Steven Pavletic ,&nbsp;Christine N. Duncan ,&nbsp;Seth J. Rotz ,&nbsp;Kirsten Williams ,&nbsp;Paul A. Carpenter ,&nbsp;Geoffrey D.E. Cuvelier","doi":"10.1016/j.jtct.2024.12.011","DOIUrl":"10.1016/j.jtct.2024.12.011","url":null,"abstract":"<div><div>Current literature lacks details on the impact of pediatric chronic graft-versus-host disease (cGVHD) on long-term survivorship after allogeneic hematopoietic cell transplantation (HCT). Nonetheless, cGVHD remains a leading cause of post-transplant morbidity and mortality in children and adolescents, which is particularly relevant given the longer life-expectancy after HCT (measured in decades) compared to older adults. To address this knowledge gap, leaders of the Pediatric Transplant and Cellular Therapy Consortium convened a multidisciplinary taskforce of experts in pediatric cGVHD and HCT late effects known as RESILIENT after Chronic GVHD (Research and Education towards Solutions for Late effects to Innovate, Excel, and Nurture after cGVHD). Our goals were to define: (1) the current state of understanding about how cGVHD impacts long-term survivorship in children transplanted &lt;18 yr of age; (2) practical aspects of care to help clinicians managing long-term pediatric cGVHD survivors; and (3) develop a research framework for the next decade to further our knowledge. Four working groups were formed, each tasked with addressing a unique theme: (1) cGVHD natural history (phases of cGVHD) and its impact on clinicians’ ability to taper and durably discontinue systemic therapy; (2) organ dysfunction and immune reconstitution in relation to survivorship; (3) how cGVHD and its treatment impact growth, metabolism, and development in children; and (4) psychosocial health and patient reported outcomes. The 4 groups met before the 2024 BMT Tandem Meeting in San Antonio, Texas, and then convened a larger in-person RESILIENT conference held on February 20, 2024, at the Tandem meeting to put forth recommendations from their respective working groups and garner feedback. These recommendations are now presented in a series of 4 manuscripts. This current manuscript focuses on the first theme and discusses the phases of cGVHD, challenges in differentiating clinically active from quiescent cGVHD in clinical practice, and the resultant difficulties in determining when and if to taper systemic therapy. To overcome these challenges, we propose revised categorization of long-term cGVHD outcomes and practical recommendations for clinicians and researchers around the long-term follow-up for these patients, including determining when and if to taper systemic therapy, along with the integration of non-immunosuppressive supportive care interventions.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 69.e1-69.e18"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865584","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of Thrombopoietin Receptor Agonists Plus Recombinant Human Thrombopoietin versus Recombinant Human Thrombopoietin Alone for Hematopoietic Reconstruction in Multiple Myeloma Patients Undergoing Autologous Hematopoietic Stem Cell Transplantation","authors":"Xuezhu Xu ,&nbsp;Ruoyu Yang ,&nbsp;Yachun Jia ,&nbsp;Gongzhizi Gao ,&nbsp;Tianyu Huang ,&nbsp;Aili He ,&nbsp;Fangxia Wang","doi":"10.1016/j.jtct.2024.12.015","DOIUrl":"10.1016/j.jtct.2024.12.015","url":null,"abstract":"<div><h3>Background</h3><div>Hetrombopag is a novel thrombopoietin receptor agonist that has shown an additive effect in stimulating platelet production when combined with recombinant human thrombopoietin (rhTPO). However, it remains unclear whether this combination can promote hematopoietic reconstruction after autologous stem cell transplant (ASCT).</div></div><div><h3>Purpose</h3><div>To compare the effect of rhTPO plus thrombopoietin receptor agonists (TPO-RA) versus rhTPO alone on hematopoietic recovery, adverse events, postoperative complications, and cost-effectiveness in patients with newly diagnosed multiple myeloma (NDMM) undergoing ASCT.</div></div><div><h3>Methods</h3><div>A total of 67 consecutive NDMM patients who underwent ASCT at our hospital from January 2021 to May 2024 were included. Of these patients, 35 received a combination of rhTPO and the TPO-RA hetrombopag after stem cell reinfusion (observation group), whereas 32 patients received rhTPO alone (control group). Hematopoietic reconstitution between the two groups was compared.</div></div><div><h3>Results</h3><div>Baseline clinical characteristics were similar between both groups. In the observation group, the median time to platelet recovery was 9 days after stem cell reinfusion, which was significantly shorter than that in the control group (<em>P</em> = .003). The mean number of platelet transfusions in the observation group was significantly lower than that in the control group (1.0 vs. 2.0 units, <em>P</em> = .034). All patients tolerated rhTPO and TPO-RA well, with no thrombotic events observed. Survival analysis showed no reduction in time to progression (TTP) and overall survival (OS) with the addition of TPO-RA. There were no statistical differences in the incidence of adverse events, drug expenses, and hospital stay between two groups (<em>P</em>  &gt; 0.05).</div></div><div><h3>Conclusions</h3><div>Although sample size and study design limit the data from this study, our findings suggest that the combination of TPO-RA (hetrombopag) and rhTPO enhances platelet recovery in comparison with rhTPO alone, without increasing adverse effects.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 84.e1-84.e8"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(25)00041-7","DOIUrl":"10.1016/S2666-6367(25)00041-7","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Page A6"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143168672","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Looking in the Rearview as We Drive CARs Forward","authors":"Liora M. Schultz","doi":"10.1016/j.jtct.2025.01.034","DOIUrl":"10.1016/j.jtct.2025.01.034","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 63-65"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143080922","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Systematic Review and Meta-Analysis of Extracorporeal Photopheresis for the Treatment of Steroid-Refractory Chronic Graft-Versus-Host Disease","authors":"Zachariah DeFilipp ,&nbsp;Laura Fox ,&nbsp;Tobias A.W. Holderried ,&nbsp;Varun Mehra ,&nbsp;David Michonneau ,&nbsp;Alex Pashley ,&nbsp;Andrei Karlsson ,&nbsp;Dennis Dong Hwan Kim","doi":"10.1016/j.jtct.2024.11.004","DOIUrl":"10.1016/j.jtct.2024.11.004","url":null,"abstract":"<div><div>The objective of this meta-analysis (MA) was to evaluate the efficacy and safety of extracorporeal photopheresis (ECP) for the treatment of steroid-refractory chronic graft-versus-host disease (SR-cGvHD). A systematic literature review (SLR) was conducted according to PRISMA guidelines, followed by a feasibility assessment (FA) to assess potential between-study heterogeneity in the meta-analysis (MA). Random-effects MAs were performed for overall survival (OS), failure-free survival (FFS), overall response rate (ORR) and skin-specific response. A subgroup analysis was conducted to explore the effect of NIH assessment criteria. The SLR identified 627 records; 45 unique studies were ultimately included in the MA. For patients treated with ECP, at Month 12, the pooled OS rate was 83.97% and the pooled FFS rate was 60.79%. ORR was 45.34% at Months 3 to 4 and 58.23% at Months 6 to 8. Subgroup analyses showed no significant difference in ORR between studies utilizing NIH criteria and those utilizing non-NIH criteria. Skin-specific response was 34.86% at Months 2 to 3 and 54.22% at Months 4 to 6. There was considerable heterogeneity across all analyses, with I² values ranging from 65% to 91%. This SLR and MA indicates that ECP results in favorable outcomes in the treatment of SR-cGvHD, including OS, FFS and ORR.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 76.e1-76.e13"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142628947","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}